Allele-Specific Gene Editing to Rescue Dominant CRX-Associated LCA7 Phenotypes in a Retinal Organoid Model

Investigators generated LCA7 retinal organoids harboring a dominant disease-causing mutation in CRX. The LCA7 retinal organoids developed signs of immature and dysfunctional photoreceptor cells, providing a reliable in vitro model to recapitulate LCA7.